About RTW Foundation (RTWF)

RTW Foundation (RTWF) was founded in 2018 as the philanthropic arm of RTW Investments, LP a biotech investment firm. RTWF has two major focuses: improving health equity in New York City and advancing ultra-rare disease research. Our overarching goal is to improve the health of underserved communities, whether that be from historically underinvested communities in New York City (including communities of color and migrant populations) to patients and families facing ultra-rare diseases.

Role Overview

The Research Associate, Scientific Advising, will support the Rare Disease Advising Program (RDAP) at RTW Foundation by conducting in-depth literature reviews, analyzing pre-clinical and clinical research, and synthesizing key insights to inform strategic research roadmaps, disease concept models, and therapeutic development strategies for rare diseases. This role will collaborate with RDAP foundation partners to evaluate research grant proposals, provide scientific recommendations, and contribute to the development of innovative strategies to advance rare disease research. The ideal candidate has a biomedical sciences background, experiences in rare disease research, and familiarity with therapeutic development principles. This position requires a highly analytical and detail-oriented scientist with the ability to translate complex scientific data into actionable insights for research and funding decisions.

Why Join Us?

This role offers a unique opportunity to bridge scientific research, therapeutic development, and strategic advising in the rare disease space. At RTW Foundation, you’ll play a direct role in shaping research strategies that impact patients and families facing ultra-rare diseases. If you're passionate about applying your scientific expertise to drive meaningful change, we encourage you to apply.

Key Responsibilities

Scientific Advising & Research Strategy:

• Conduct targeted literature reviews and landscape analyses to identify key challenges, knowledge gaps, and potential therapeutic opportunities for rare diseases.
• Evaluate preclinical and clinical research to inform translational strategies and therapeutic roadmaps for partnered rare disease foundations.
• Identify key researchers, institutions, and industry players working within specific rare diseases, including past and ongoing drug development efforts.
• Collaborate with the Director of Research to develop comprehensive research roadmaps aligned with therapeutic feasibility and foundation objectives.
• Track and analyze emerging therapeutic modalities (e.g., gene therapy, RNA-based therapies, gene-editing, biologics, small molecules) relevant to rare diseases.
• Identify opportunities to expand and scale the scientific advising program to better serve the rare disease community.

Grant & Research Funding Support:

• Contribute to the strategic review of research grants, ensuring alignment with cutting-edge rare disease research and translational potential.
• Engage with foundation partners and research teams to refine funding priorities based on scientific and therapeutic feasibility.
• Focus on improving grant application and review processes.

Operations & Cross-functional Support:

• Maintain research documentation and contribute to reports, presentations, and white papers for stakeholders.
• Support CRM and grant tracking systems (e.g., Salesforce) to ensure accurate records of research initiatives and funding activities.
• Participate in internal and external scientific meetings, representing RDAP and contributing to discussions on rare disease research.

Qualifications and Skills

• PhD (preferred) in biomedical sciences, genetics, pharmacology, immunology, neuroscience, or related fields, BS/MS with significant experience also considered.
• Experience in rare disease research (e.g., genetic disorders, translational research, biomarker discovery, or therapeutic development).
• Understanding of therapeutic development principles, including target validation, preclinical models, or clinical trial design.
• Familiarity with drug development pipelines and regulatory considerations (e.g., FDA, EMA, orphan drug designation) is a plus.
• Prior experience in scientific advising, grant evaluation, or non-profit/biotech research strategy is advantageous.
• Highly analytical and detail-or